Rare opportunity

Inside the world of rare diseases with Joe Wiley, CEO of Amryt Pharma

Currently I am chief executive of Amryt Pharma, which I co-founded two years ago. I have over 20 years’ experience across the medical, pharmaceutical and venture capital industries.

What does your day-to-day work involve?

Each day varies greatly, but I am primarily focused on building and leading a talented team who can ensure Amryt delivers on its mission, to deliver treatments for people living with rare and very debilitating conditions. I also invest a lot of time in ensuring our partnerships are looked after and in finding potential future collaborations.

Another key element of my job is ensuring that we have a pipeline to support our ambitions and an investment level which allows us to offer potentially life-changing medicines to those living with and affected by rare diseases.

At Amryt, we’re ambitious and believe we have the skills and expertise to help to change lives.

Why the interest in rare diseases?

From my medical training many years ago while at Trinity College in Dublin, and my many years in the world of pharma, I know all too well that while we have made great strides in many areas of medicine and health outcomes, we have yet to see that kind of improvement in rare diseases. There is still much to do to be able to offer the many people living with rare conditions transformative treatment options.

While a lot in the pharma industry choose not to invest in rare diseases, we at Amryt are exclusively focused on these conditions.

What are the key issues for firms focusing on medicines for such small patient populations?

It can be very challenging to conduct clinical trials in rare diseases where trials may not have been conducted before both in terms trial design but also in terms of recruitment.

We are a small organisation and it can be tough to manage the multiple hurdles to bring innovations to patients, to get products approved by regulators and subsequently reimbursed by governments around the world.

Do you believe the new system of appraising medicines for rare diseases for NHS use will have any impact on patient access?

It remains to be seen how evolving systems for appraising medicines in the rare disease space will work in practice and whether they will deliver on making timely, flexible decisions to provide access to innovative medicines for those living with rare diseases.

We need to work in partnership to ensure we have a collective system that provides equitable access, regardless of disease type. Whilst I recognise the ambition has been to create a system that allows for access to medicines, I have concerns that we are still seeing high hurdles and long delays in decision-making in the rare disease space. Since data collection, is by definition, very challenging due to small patient populations, there needs to be an appropriate balance struck.

We need to encourage pharma companies to invest in research and development in rare diseases and without fair, timely reimbursement, we are in danger of stifling innovation and leaving these patients behind.

What do you think will be the key challenges/opportunities for Amryt posed by Brexit?

We will have to decide where to hold our marketing authorisations in the EU in the future and to trust that the EMA in its new location will ensure seamless continuity in the important work that it does. I hope the UK will continue to be aligned to harmonised approval procedures.

I would like to see the continued ability for talented staff to enjoy free movement and I see there is a great opportunity for the UK, if it wants to compete on the world stage, to better align its industrial strategy to its healthcare innovation and access strategy to ensure it is attractive place for research and to conduct business in the future.

To gain approval for our lead development compound which is currently in a global phase III pivotal study, a treatment for epidermolysis bullosa which is a disfiguring and painful condition that causes the skin to blister and tear at the slightest touch.

Additionally, we plan to grow our product portfolio and global footprint to be able to bring transformative medicines to the patients who need them most.

What do you feel has been your greatest achievement to date?

Alongside focusing on delivering new treatments my greatest achievement has been to build a highly talented team who would be the envy of many companies in this arena. I’m very proud of our team who strive every day to transform the lives of people with rare, debilitating conditions. Each member of the team is empowered to be able to positively impact on the lives of others.

What are your passions outside of work?

I love to spend time with my family and I have a passion for golf, frustrating as it can be sometimes!